Benefits of glucocorticoids in non-ambulant boys/men with Duchenne muscular dystrophy: A multicentric longitudinal study using the Performance of Upper Limb test

Marika Pane, Lavinia Fanelli, Elena Stacy Mazzone, Giorgia Olivieri, Adele D'Amico, Sonia Messina, Marianna Scutifero, Roberta Battini, Roberta Petillo, Silvia Frosini, Serena Sivo, Gian Luca Vita, Claudio Bruno, Tiziana Mongini, Elena Pegoraro, Roberto De Sanctis, Alice Gardani, Angela Berardinelli, Valentina Lanzillotta, Adelina CarlesiEmanuela Viggiano, Filippo Cavallaro, Maria Sframeli, Luca Bello, Andrea Barp, Flaviana Bianco, Serena Bonfiglio, Enrica Rolle, Concetta Palermo, Grazia D'Angelo, Antonella Pini, Elena Iotti, Ksenija Gorni, Giovanni Baranello, Enrico Bertini, Luisa Politano, Maria Pia Sormani, Eugenio Mercuri

Research output: Contribution to journalArticle

24 Citations (Scopus)

Abstract

The aim of this study was to establish the possible effect of glucocorticoid treatment on upper limb function in a cohort of 91 non-ambulant DMD boys and adults of age between 11 and 26 years.All 91 were assessed using the Performance of Upper Limb test. Forty-eight were still on glucocorticoid after loss of ambulation, 25 stopped steroids at the time they lost ambulation and 18 were GC naïve or had steroids while ambulant for less than a year.At baseline the total scores ranged between 0 and 74 (mean 41.20). The mean total scores were 47.92 in the glucocorticoid group, 36 in those who stopped at loss of ambulation and 30.5 in the naïve group (p <0.001).The 12-month changes ranged between -20 and 4 (mean -4.4). The mean changes were -3.79 in the glucocorticoid group, -5.52 in those who stopped at loss of ambulation and -4.44 in the naïve group. This was more obvious in the patients between 12 and 18 years and at shoulder and elbow levels.Our findings suggest that continuing glucocorticoids throughout teenage years and adulthood after loss of ambulation appears to have a beneficial effect on upper limb function.

Original languageEnglish
Pages (from-to)749-753
Number of pages5
JournalNeuromuscular Disorders
Volume25
Issue number10
DOIs
Publication statusPublished - Oct 1 2015

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Duchenne Muscular Dystrophy
Upper Extremity
Glucocorticoids
Walking
Longitudinal Studies
Steroids
Elbow

Keywords

  • Duchenne muscular dystrophy
  • Glucocorticoids
  • Non ambulant
  • PUL
  • Upper limb

ASJC Scopus subject areas

  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health
  • Genetics(clinical)
  • Neurology

Cite this

Benefits of glucocorticoids in non-ambulant boys/men with Duchenne muscular dystrophy : A multicentric longitudinal study using the Performance of Upper Limb test. / Pane, Marika; Fanelli, Lavinia; Mazzone, Elena Stacy; Olivieri, Giorgia; D'Amico, Adele; Messina, Sonia; Scutifero, Marianna; Battini, Roberta; Petillo, Roberta; Frosini, Silvia; Sivo, Serena; Vita, Gian Luca; Bruno, Claudio; Mongini, Tiziana; Pegoraro, Elena; De Sanctis, Roberto; Gardani, Alice; Berardinelli, Angela; Lanzillotta, Valentina; Carlesi, Adelina; Viggiano, Emanuela; Cavallaro, Filippo; Sframeli, Maria; Bello, Luca; Barp, Andrea; Bianco, Flaviana; Bonfiglio, Serena; Rolle, Enrica; Palermo, Concetta; D'Angelo, Grazia; Pini, Antonella; Iotti, Elena; Gorni, Ksenija; Baranello, Giovanni; Bertini, Enrico; Politano, Luisa; Sormani, Maria Pia; Mercuri, Eugenio.

In: Neuromuscular Disorders, Vol. 25, No. 10, 01.10.2015, p. 749-753.

Research output: Contribution to journalArticle

Pane, M, Fanelli, L, Mazzone, ES, Olivieri, G, D'Amico, A, Messina, S, Scutifero, M, Battini, R, Petillo, R, Frosini, S, Sivo, S, Vita, GL, Bruno, C, Mongini, T, Pegoraro, E, De Sanctis, R, Gardani, A, Berardinelli, A, Lanzillotta, V, Carlesi, A, Viggiano, E, Cavallaro, F, Sframeli, M, Bello, L, Barp, A, Bianco, F, Bonfiglio, S, Rolle, E, Palermo, C, D'Angelo, G, Pini, A, Iotti, E, Gorni, K, Baranello, G, Bertini, E, Politano, L, Sormani, MP & Mercuri, E 2015, 'Benefits of glucocorticoids in non-ambulant boys/men with Duchenne muscular dystrophy: A multicentric longitudinal study using the Performance of Upper Limb test', Neuromuscular Disorders, vol. 25, no. 10, pp. 749-753. https://doi.org/10.1016/j.nmd.2015.07.009
Pane, Marika ; Fanelli, Lavinia ; Mazzone, Elena Stacy ; Olivieri, Giorgia ; D'Amico, Adele ; Messina, Sonia ; Scutifero, Marianna ; Battini, Roberta ; Petillo, Roberta ; Frosini, Silvia ; Sivo, Serena ; Vita, Gian Luca ; Bruno, Claudio ; Mongini, Tiziana ; Pegoraro, Elena ; De Sanctis, Roberto ; Gardani, Alice ; Berardinelli, Angela ; Lanzillotta, Valentina ; Carlesi, Adelina ; Viggiano, Emanuela ; Cavallaro, Filippo ; Sframeli, Maria ; Bello, Luca ; Barp, Andrea ; Bianco, Flaviana ; Bonfiglio, Serena ; Rolle, Enrica ; Palermo, Concetta ; D'Angelo, Grazia ; Pini, Antonella ; Iotti, Elena ; Gorni, Ksenija ; Baranello, Giovanni ; Bertini, Enrico ; Politano, Luisa ; Sormani, Maria Pia ; Mercuri, Eugenio. / Benefits of glucocorticoids in non-ambulant boys/men with Duchenne muscular dystrophy : A multicentric longitudinal study using the Performance of Upper Limb test. In: Neuromuscular Disorders. 2015 ; Vol. 25, No. 10. pp. 749-753.
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AU - Fanelli, Lavinia

AU - Mazzone, Elena Stacy

AU - Olivieri, Giorgia

AU - D'Amico, Adele

AU - Messina, Sonia

AU - Scutifero, Marianna

AU - Battini, Roberta

AU - Petillo, Roberta

AU - Frosini, Silvia

AU - Sivo, Serena

AU - Vita, Gian Luca

AU - Bruno, Claudio

AU - Mongini, Tiziana

AU - Pegoraro, Elena

AU - De Sanctis, Roberto

AU - Gardani, Alice

AU - Berardinelli, Angela

AU - Lanzillotta, Valentina

AU - Carlesi, Adelina

AU - Viggiano, Emanuela

AU - Cavallaro, Filippo

AU - Sframeli, Maria

AU - Bello, Luca

AU - Barp, Andrea

AU - Bianco, Flaviana

AU - Bonfiglio, Serena

AU - Rolle, Enrica

AU - Palermo, Concetta

AU - D'Angelo, Grazia

AU - Pini, Antonella

AU - Iotti, Elena

AU - Gorni, Ksenija

AU - Baranello, Giovanni

AU - Bertini, Enrico

AU - Politano, Luisa

AU - Sormani, Maria Pia

AU - Mercuri, Eugenio

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